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Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - PAH-HOPE (New hope for the PAH patient: A Novel Therapeutic for Pulmonary Arterial Hypertension (PAH))

Teaser

Summary

Pulmonary arterial hypertension (PAH) is a debilitating and life-threatening disease that begins in the lungs of the PAH patient but can also rapidly affect the heart, resulting in heart failure and death. The disease occurs in people at any age and is 3-4 times more prevalent in women than men. Despite being classed as a rare or orphan disease, affecting 15-50 patients in a million, PAH carries an enormous health burden globally. While current drugs can slow the rate at which the disease advances and extend life expectancy, they are less than effective in treating most aspects of the disease. Moreover, many of the current therapies are poorly tolerated and carry serious side-effects. Thus, in the opinion of PAH patients, treating physicians and healthcare payors, there is â€œan urgent medical need for new drugs to new drug targets which treat all aspects of the diseaseâ€.
ATXA Therapeutics Ltd (ATXA), a biotech company based in Dublin (Ireland), has developed novel therapeutic drugs targeting an entirely unique pathway that treats all hallmark causes and symptoms of PAH. Based on evidence from animal models of the disease, ATXAâ€™s lead NTP42 is predicted to be a truly disruptive disease-modifying drug, treating all aspects of PAH and without carrying any serious side-effects. As an external endorsement of this, ATXA has secured orphan designation from the main regulatory authorities in Europe (the European Medicines Agency/ EMA) and in the USA (the Food and Drugs Administration/ FDA) for use of NTP42 in PAH patients following market launch.
Thus, through this exciting opportunity afforded to ATXA by the H2020 SME Instrument-funding mechanism, the mission and overarching objective of PAH-HOPE is to bring new hope to the PAH patient by advancing NTP42 into clinical development, starting with scaled up drug manufacture through to securing regulatory clinical trial authorization (CTA). Securing CTA will be a major milestone of PAH-HOPE, positioning ATXA to advance NTP42 to Phase I-III clinical trial testing in humans through to marketing authorization and product launch in 2025, thereby solving the urgent unmet medical need of the PAH patient, treating physician, healthcare payor and market. Through an ambitious series of technical, regulatory and business-led activities or work packages, involving a highly skilled and dedicated team, PAH-HOPE is on course to deliver on its mission and overall objective.

Work performed

To achieve this objective, starting with Drug Substance Manufacture, the PAH-HOPE team have successfully completed the scale-up manufacture generating high quality pharmaceutical grade NTP42 that exceeds pre-approved specifications while also developing a unique and highly efficient, robust manufacturing process. Following on from scale-up manufacture, the next steps are to formulate NTP42 into a form that is suitable for use as an oral medicine (Formulation and Packaging) as well as to test it in formal drug safety (Drug Safety & Toxicology) studies using guidelines specified by the EMA and FDA regulatory authorities. The PAH-HOPE team have made substantial progress in developing a good oral formulation for NTP42 that is easily manufactured and which remains stable on storage over time. Furthermore, the team have finalized the formal drug safety-testing package with several activities commenced, while others will be carried out during the next reporting period coinciding with completion of NTP42 medicinal formulation. Thus, through these 3 technical activities or work packages, there has been substantial progress in meeting the overall objective of PAH-HOPE. In parallel with this, successful completion of the scale-up manufacture of NTP42 also generated key regulatory compliance reports and certificates required for filing of key chapters or activities generally associated with Regulatory Authorization, including for preparation of the CTA application. In turn, equivalent reports and certificates validating the formulation and safety of NTP42 will be generated and incorporated into the CTA for regulatory filing. Furthermore, from a commercial and business perspective (IP and Commercialization Plan), the achievements of these technical milestones have generated a significant amount of new intellectual property (IP), resulting in expansion of ATXAâ€™s robust patent estate through new patent filings. Moreover, as ATXA and the PAH-HOPE management team advance the business and commercial objectives of PAH-HOPE, during the first reporting period, a robust, yet adaptable business plan has been generated along with other supporting business and/or promotional material to meet the demands of current and future investors. Furthermore, the scientific and management team have made substantial progress in activities generally associated with Engagement & Communication, disseminating the mission, objectives, results and achievements of PAH-HOPE to all stakeholders, be they the PAH patient, our ultimate customer, key opinion leaders (KOLs) expert in pharmaceutical development, business strategy, fundraising or patient treatment. This has involved a variety of platforms, be it through scheduled face-to-face meetings, attendance or podium presentations at clinical or investor conferences, press releases, media coverage, formal peer-review manuscript publications or through on-line portals such as the dedicated PAH-HOPE website (https://www.atxatherapeutics.com/pah-hope), social media or video (https://youtu.be/Um0QEQwiY4A), and has resulted in significant outreach and positive feedback while socializing the benefits of NTP42 for the PAH patient and the overarching ambition of ATXA and PAH-HOPE. Each of these activities and achievements have been made possible by ensuring excellence in Governance and Management of the PAH-HOPE program at ATXA, instigated through effective internal and external communication and reporting structures between all parties be they dedicated staff, scientific or clinical advisors or specialist sub-contractors, as well as putting in place a secure and efficient IT infrastructure.

Final results

PAH is a serious clinical diagnosis. Current therapies are failing. What the patient needs is new treatments in the form of oral medications that will modify the course of the disease, preventing or, even better, reversing its progression. Through the achievements of PAH-HOPE, ATXA is on-target to deliver on its promise to the PAH patient by bringing to the market an entirely first-in-class medicinal entity, namely NTP42, that will treat all aspects of PAH and without the side-effects associated with existing medications. As the company achieves this objective of bringing its disruptive innovation to the marketplace, ATXA also intends to be a top-tier pharmaceutical leader in PAH, treating patients across Europe and globally while addressing the large socio-economic burden for the patient and payor. Furthermore, while PAH is a rare or orphan disease, its causes and symptoms occur in several other highly prevalent diseases, many of which are also life threatening and carry heavy socio-economic burdens in Europe and globally. Hence, the possible commercial market and socio-economic impact and benefits of NTP42 as a therapeutic drug to the wider community may far extend beyond that of PAH.